Assessing a new treatment for Alpha-1 Antitrypsin Deficiency
A Phase 1b/2a Open-label Single Ascending Doses (SAD) and Multiple Ascending Doses (MAD) Research Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Participants with AATD Pi*ZZ on WVE-006 (RestorAATion-2)
This study is testing a new treatment called WVE-006 to see if it is safe and effective for people with Alpha-1 Antitrypsin Deficiency who have mild to moderate lung or liver issues.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 24 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | Wave Life Sciences Ltd. Industry-sponsored |
| Locations | 6 sites (Fitzroy, Victoria and 5 other locations) |
| Trial ID | NCT06405633 on ClinicalTrials.gov |
What this trial studies
This open-label study evaluates the safety and tolerability of WVE-006 in participants with Alpha-1 Antitrypsin Deficiency (AATD) through both single and multiple ascending doses. The study aims to understand the pharmacodynamics and pharmacokinetics of the treatment in individuals with mild to moderate AATD-induced lung or liver disease. Participants will be monitored for their response to the treatment and any adverse effects that may arise during the dosing periods.
Who should consider this trial
Good fit: Ideal candidates include individuals with confirmed Pi*ZZ genotype and mild to moderate AATD-induced lung or liver disease.
Not a fit: Patients with severe AATD or those with significant comorbidities that interfere with treatment may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from Alpha-1 Antitrypsin Deficiency.
How similar studies have performed: While there have been studies on AATD treatments, this specific approach using WVE-006 is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Healthy as determined by the Investigator, based on a medical evaluation, or has mild to moderate AATD-induced lung disease (FEV1 ≥50%) and/or stable mild AATD-induced liver disease (≤F2 (≤10 kPa) on FibroScan. * Genetic testing confirming Pi\*ZZ. * Participant has been a non-smoker for at least 1 year prior to screening. Exclusion Criteria: * • Participant has a history of multiple drug allergies or of allergic reaction to an oligonucleotide or to N-acetylgalactosamine (GalNAc). * Participant has a history of intolerance or any medical condition that might interfere with subcutaneous injections. * Any ongoing or recent infections. * Any recent or planned vaccinations during the study. * Participant has a history of regular alcohol consumption exceeding 14 standard drinks/week. * Unwilling to abstain from alcohol for 48 hours prior to dosing at each of the dosing visits. * Any recent or planned major surgery during the study. * Participant has any medical condition or social circumstance that, in the opinion of the Investigator, would make the participant unsuitable for participation in the study or for dosing on Day 1, or could interfere with the assessments of safety, pharmacodynamics, or pharmacokinetics, or completion of the study. * Participant currently on AAT augmentation therapy, planned to be on augmentation therapy anytime during the study, or has been on augmentation therapy within 30 days prior to Screening Visit. * Donation of blood or blood products in excess of 500 mL within 12 weeks prior to Screening Visit and/or unwilling to refrain from blood donation for the duration of the study. * Participant has received an investigational agent within 3 months of the Screening Visit.
Where this trial is running
Fitzroy, Victoria and 5 other locations
- St. Vincent's Hospital, Melbourne — Fitzroy, Victoria, Australia (Recruiting)
- Dalhousie University - Queen Elizabeth II Health Sciences Centre — Halifax, Nova Scotia, Canada (Recruiting)
- Inspiration Research Limited — Toronto, Ontario, Canada (Recruiting)
- Turku University Hospital — Turku, Finland (Recruiting)
- Waikato Hospital — Hamilton, New Zealand (Recruiting)
- Queen Elizabeth Hospital, University Hospital Birmingham — Birmingham, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Clinical Operations
- Email: Clinicaltrials@wavelifesci.com
- Phone: 855-215-4687
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.